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FDA Breakthrough Therapy Designation: GSK Receives Approval For Additional Indication For Promacta

August 26, 2014

GlaxoSmithKline (GSK) announces on August 26th that the  FDA approves a supplemental New Drug Application (sNDA), for the once-daily use of Promacta (Eltrombopag) in patients with Severe Aplastic Anemia (SAA), who have had an insufficient response to Immunosuppressive Therapy (IST). Promacta is a new 1st-in-class treatment option for previously treated SAA patients.

Eltrombopag is marketed under the brand name Promacta in the US and Revolade in most ex-US countries. In addition to the approval of Promacta for SAA in the US, eltrombopag is indicated for the treatment of thrombocytopenia in patients.

SAA is a rare disorder where the bone marrow fails to make enough new blood cells. There are currently no therapies approved for this indication. About forty percent (40%) of patients who do not respond to initial IST die within 5 years of diagnosis.

FDA Regulatory Actions

•   Orphan Drug Designation (ODD) in May 2008 & approval in November 2008 for Thrombocytopenia

•   ODD in November 2013 for Aplastic Anemia

•   Breakthrough Therapy Designation (BTD) in February 2014 for cytopenias in patients with SAA who have had insufficient response to IST

•   Priority Review in April 2014

•   Approval for additional indication in August 2014 for patients with SAA who have had insufficient response to IST.



FDA BTD Approval Chart

FDA BTD Statistics Chart.

Please Note: “Icon Announcer” by Orion 8 [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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